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Adeno-Associated Viral Vector Serotype 9 Containing The Human Habcd1 Gene

This article provides an overview of a clinical trial investigating the use of SBT101, an adeno-associated viral vector serotype 9 containing the human HABCD1 gene, as a potential gene therapy for adults with adrenomyeloneuropathy (AMN). The study aims to evaluate the safety, tolerability, and efficacy of this innovative treatment approach for a rare genetic disorder affecting the nervous system.

Table of Contents

What is SBT101?

SBT101 is a new type of medicine called gene therapy. It’s being developed to treat a rare genetic condition called Adrenomyeloneuropathy (AMN). SBT101 is made by a company called SwanBio Therapeutics, Ltd.[1]

This medicine is special because it’s not a pill or injection like most medicines. Instead, it uses a modified virus to deliver a healthy copy of a gene that’s not working properly in people with AMN. The scientific name for SBT101 is “adeno-associated viral vector serotype 9 containing the human ABCD1 gene.”[1]

How Does SBT101 Work?

SBT101 works by using a harmless virus called AAV9 to carry a healthy copy of the ABCD1 gene into the body. In people with AMN, this gene doesn’t work correctly. By providing a working copy of the gene, SBT101 aims to help the body produce a protein that’s missing in AMN patients.[1]

The medicine is given through a procedure called “intrathecal administration“. This means it’s injected directly into the fluid surrounding the spinal cord. This method is chosen to help the medicine reach the nervous system where it’s needed most.[1]

What Condition Does SBT101 Treat?

SBT101 is designed to treat Adrenomyeloneuropathy (AMN). AMN is a rare genetic disorder that affects the spinal cord and nerves. It’s a type of X-linked adrenoleukodystrophy (ALD). People with AMN have difficulty walking, problems with balance, and may experience other nervous system issues.[1]

AMN is caused by mutations in the ABCD1 gene. This gene helps break down certain fatty substances in the body. When it doesn’t work properly, these fatty substances build up and damage the protective coating around nerves, leading to the symptoms of AMN.[1]

Clinical Trial Details

SBT101 is currently being tested in a clinical trial. This trial is a Phase 1/2 study, which means it’s an early stage of testing in humans. The main goals of this study are:[1]

  1. To check if SBT101 is safe for patients with AMN
  2. To see how well SBT101 works in treating AMN

The trial will test different doses of SBT101 to find the best and safest dose. Patients will be randomly assigned to receive either SBT101 or a placebo (a treatment with no active medicine). Neither the patients nor the doctors will know who is receiving which treatment until the end of the study. This type of study is called “randomized” and “blinded”.[1]

Who Can Participate in the SBT101 Trial?

The trial is looking for specific types of patients. Here are some of the main criteria:[1]

  • Men aged 18 to 65 years old
  • Diagnosed with X-linked adrenoleukodystrophy (ALD) with a confirmed mutation in the ABCD1 gene
  • Show signs of spinal cord involvement, but not severe disability
  • No signs of brain inflammation related to ALD
  • No other serious health conditions that could interfere with the study

There are also several reasons why someone might not be able to participate, such as having certain other medical conditions or having received gene therapy before.[1]

What to Expect During the Trial

If you participate in the trial, here’s what you might expect:[1]

  • A one-time treatment with SBT101 (or placebo) given through a spinal injection
  • Regular check-ups and tests over 24 months, including walking tests, balance tests, and questionnaires about how you’re feeling
  • MRI scans of your brain
  • Blood tests and other medical exams

The researchers will be looking at several things, including how far you can walk in 6 minutes, how quickly you can stand up from a sitting position, and how your balance changes over time.[1]

Potential Benefits and Risks

As with any new treatment, there are potential benefits and risks to consider:[1]

Potential benefits:

  • SBT101 might slow down or stop the progression of AMN symptoms
  • It could improve walking ability, balance, and overall quality of life
  • Participating in the trial contributes to medical research that could help many people in the future

Potential risks:

  • There might be side effects from the treatment, which are not yet fully known
  • The treatment might not work as well as hoped
  • There are risks associated with the spinal injection procedure

It’s important to discuss these potential benefits and risks thoroughly with your doctor before deciding to participate in any clinical trial.

Aspect Details
Study Type Phase 1/2 Randomized, Blinded, Dose-escalation Study
Intervention SBT101 (AAV9-ABCD1 Gene Therapy)
Administration Intrathecal (one-time)
Target Population Adult males (18-65 years) with Adrenomyeloneuropathy
Primary Endpoint Safety and tolerability at 24 months
Key Secondary Endpoints Changes in 6MWT, 2MWT, 5XSST, EDSS, and other functional tests at 12 and 24 months
Long-term Follow-up Up to 60 months for safety assessment

FAQ

  • What is SBT101 and how does it work? SBT101 is a gene therapy that uses an adeno-associated viral vector serotype 9 to deliver the human HABCD1 gene. It is designed to treat adrenomyeloneuropathy by potentially correcting the genetic defect responsible for the condition.
  • Who is eligible for this clinical trial? The trial is open to male adults aged 18-65 years with a confirmed diagnosis of X-linked adrenoleukodystrophy, including a proven mutation in the ABCD1 gene and clinical evidence of spinal cord involvement.
  • How is SBT101 administered in this trial? SBT101 is administered intrathecally, which means it is injected directly into the spinal fluid. This is a one-time treatment in the context of this clinical trial.
  • What are the main objectives of this clinical trial? The primary objective is to evaluate the safety and tolerability of SBT101 in adults with AMN. Secondary objectives include assessing its efficacy through various physical and neurological tests.
  • How long will participants be monitored in this trial? Participants will be monitored for at least 24 months, with some endpoints being evaluated up to 60 months after treatment to assess long-term safety and efficacy.

Ongoing Clinical Trials on Adeno-Associated Viral Vector Serotype 9 Containing The Human Habcd1 Gene

  • Study on the Safety and Effectiveness of SBT101 Gene Therapy for Adults with Adrenomyeloneuropathy

    Not recruiting

    1 1
    Investigated drugs:
    The Netherlands

Glossary

  • Adrenomyeloneuropathy (AMN): A rare genetic disorder affecting the spinal cord and nerves, caused by mutations in the ABCD1 gene. It primarily affects adult males and leads to progressive neurological symptoms.
  • Gene therapy: A treatment approach that involves introducing genetic material into a person's cells to compensate for abnormal genes or to make a beneficial protein.
  • Adeno-Associated Viral Vector (AAV): A virus modified for use in gene therapy to deliver genetic material to cells without causing disease.
  • Intrathecal administration: A method of delivering medication directly into the spinal fluid surrounding the brain and spinal cord.
  • ABCD1 gene: The gene responsible for producing a protein involved in the breakdown of very long-chain fatty acids. Mutations in this gene cause X-linked adrenoleukodystrophy and adrenomyeloneuropathy.
  • Expanded Disability Status Scale (EDSS): A method of quantifying disability in multiple sclerosis and similar neurological conditions, including adrenomyeloneuropathy.
  • 6-minute Walk Test (6MWT): A test that measures the distance an individual can walk in 6 minutes, used to assess functional capacity and endurance.
  • Loes score: A system used to quantify the severity of brain abnormalities on MRI scans in patients with adrenoleukodystrophy.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-sbt101-gene-therapy-for-adults-with-adrenomyeloneuropathy/